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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06849973
Registration number
NCT06849973
Ethics application status
Date submitted
24/02/2025
Date registered
25/03/2025
Date last updated
25/03/2025
Titles & IDs
Public title
To Study the Efficacy & Safety of Oral NA-921 (Bionetide) in Girls and Women with Rett Syndrome
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Scientific title
A Randomized, Double-Blind, Placebo-Controlled, Phase 2/ Phase 3 Study of NA-921 (Bionetide) for the Treatment of Girls and Women with Rett Syndrome
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Secondary ID [1]
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BIO-2024-R01
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Rett Syndrome
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Neurological
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Other neurological disorders
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Other
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Research that is not of generic health relevance and not applicable to specific health categories listed above
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Drug: Bionetide
Treatment: Drugs - Placebo
Experimental: Experimental: Drug - NA-921 (Bionetide) - NA-921 (Bionetide) solution of 20-40 mL based on the subject's weight at Basel
Placebo comparator: Placebo Comparator: Placebo - Bionetide placebo solution of 20-40 mL based on the subject's weight at Baseline, administered twice daily by mouth or gastrostomy tube (G-tube)
Treatment: Drugs: Drug: Bionetide
NA-921 (Bionetide) solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
Treatment: Drugs: Placebo
NA-921 (Bionetide) placebo solution administered based on the subject's weight at Baseline, twice daily for 12 weeks
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Rett Syndrome Behavior Questionnaire (RSBQ) Total Score - Change From Baseline to Week 12
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Assessment method [1]
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The RSBQ is a 45-item caregiver-completed rating scale that includes 45 items, 39 of them grouped into 8 subscales, whose ratings reflect the severity and frequency of symptoms. Items are rated as 0 (not true), 1 (somewhat or sometimes true), or 2 (very true). The 8 subscales are general mood, breathing problems, hand behavior, face movements, body rocking/expressionless face, night-time behaviors, fear/anxiety, and walking/standing. Scores for item 31 are reversed in the calculation of the total score. The total score ranges from 0 to 90 and is calculated as the sum of the item scores. Higher scores mean worse behavior.
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Timepoint [1]
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Baseline and Week 12
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Primary outcome [2]
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Clinical Global Impression-Improvement (CGI-I) Score at Week 12
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Assessment method [2]
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To rate how much the subject's illness has improved or worsened relative to a baseline state, a 7-point scale is used from 1=very much improved, 2=much improved, 3=minimally improved, 4=no change, 5=minimally worse, 6=much worse, 7=very much worse.
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Timepoint [2]
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12 Weeks Treatment Duration
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Secondary outcome [1]
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Change From Baseline to Week 12 in Communication and Symbolic Behavior Scales Developmental Profile™ Infant-Toddler Checklist - Social Composite Score (CSBS-DP-IT Social)
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Assessment method [1]
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Standardized screening scale for assessing communication and pre-linguistic skills in young children 12-24 months and can be used with older children with developmental delay. The CSBS-DP includes a suite of three separate measures: The Infant-Toddler Checklist, a follow-up Caregiver Questionnaire and a Behavior Sample. In this study only the Infant-Toddler (CSBS-DP-IT) Checklist was used. The CSBS-DP-IT Checklist is a 24-item rating scale and each item is scored using a three-level rating of frequency: "not yet", "sometimes" and "often". The CSBS-DP-IT Social Composite score the range was 0 to 26 and a higher score represented a worse outcome. Three composite scores can be calculated: 1) Social Composite; 2) Speech Composite; 3) Symbolic Composite.
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Timepoint [1]
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12 Weeks Treatment Duration
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Secondary outcome [2]
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Change From Baseline to Week 12 in Rett Syndrome Clinician Rating of Ability to Communicate Choices (RTT-COMC)
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Assessment method [2]
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Clinical assessment of the subject's ability to communicate their choices or preferences, which can include the use of nonverbal means such as eye contact or gestures. The assessment is made on an 8-point Likert scale (0-7) with 0 denoting normal functioning and 7 the most severe impairment.
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Timepoint [2]
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12 Weeks Treatment Duration
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Eligibility
Key inclusion criteria
INCLUSION CRITERIA:
* Female subjects 5 to 20 years of age, inclusive, at Screening
* Body weight =12 kg at Screening
* Can swallow the study medication provided as a liquid solution or can take it by gastrostomy tube
* Has classic/typical Rett syndrome (RTT)
* Has a documented disease-causing mutation in the MECP2 gene
* Has a stable pattern of seizures, or has had no seizures, within 8 weeks of Screening
* Subjects of childbearing potential must abstain from sexual activity for the duration of the study and for at least 30 days thereafter or must agree to use acceptable methods of contraception. Subject must not be pregnant or breastfeeding.
* The subject's caregiver is English-speaking and has sufficient language skills to complete the caregiver assessments
* Subject and caregiver(s) must reside at a location to which study drug can be delivered and have been at their present residence for at least 3 months prior to Screening
EXCLUSION CRITERIA :
* Has been treated with insulin within 12 weeks of Baseline
* Has current clinically significant cardiovascular, endocrine (such as hypo- or hyperthyroidism, Type 1 diabetes mellitus, or uncontrolled Type 2 diabetes mellitus), renal, hepatic, respiratory or gastrointestinal disease (such as celiac disease or inflammatory bowel disease) or has major surgery planned during the study
* Has a history of, or current, cerebrovascular disease or brain trauma
* Has significant, uncorrected visual or uncorrected hearing impairment
* Has a history of, or current, malignancy
* Has a known history or symptoms of long QT syndrome
Additional inclusion/exclusion criteria apply. Patients will be evaluated at screening to ensure that all criteria for study participation are met. Patients may be excluded from the study based on these assessments (and specifically, if it is determined that their baseline health and condition do not meet all pre-specified entry criteria).
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Minimum age
5
Years
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Maximum age
20
Years
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Sex
Females
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Can healthy volunteers participate?
No
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Key exclusion criteria
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
15/03/2024
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
28/01/2025
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Sample size
Target
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Accrual to date
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Final
187
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Recruitment in Australia
Recruitment state(s)
NSW,VIC
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Recruitment hospital [1]
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Biomed Research Unit-BIO-16-NSW - Camperdown
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Recruitment hospital [2]
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Biomed Research Unit-BIO-15 - Sydney
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Recruitment hospital [3]
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Biomed Research Unit-BIO-13-VIC-3084 Heidelberg West, Victoria, Australia, 3084 - Heidelberg West
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Recruitment hospital [4]
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Biomed Research Unit- BIO-14-VIC 3010 - Parkville
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Recruitment postcode(s) [1]
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2050 - Camperdown
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Recruitment postcode(s) [2]
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2060 - Sydney
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Recruitment postcode(s) [3]
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3084 - Heidelberg West
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Recruitment postcode(s) [4]
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3010 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Colorado
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Country [3]
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United States of America
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Illinois
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Country [4]
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United States of America
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State/province [4]
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Maryland
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Country [5]
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United States of America
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State/province [5]
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Massachusetts
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Country [6]
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United States of America
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State/province [6]
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New York
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Country [7]
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United States of America
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State/province [7]
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Ohio
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Country [8]
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United States of America
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State/province [8]
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Texas
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Country [9]
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United States of America
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State/province [9]
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Washington
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Biomed Industries, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
To study the efficacy \& safety of oral Bionetide versus placebo in girls and women with Rett syndrome
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Trial website
https://clinicaltrials.gov/study/NCT06849973
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Lloyd L. Tran, PhD
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Address
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Biomed Industries, Inc.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Phone
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Fax
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Email
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Undecided
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No/undecided IPD sharing reason/comment
We will decide whether to share IPD at a later date
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT06849973
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